FREIBURG, Germany — September 15, 2026
One04 Therapeutics GmbH & Co. KG announced its launch as a clinical-stage biopharmaceutical company following encouraging interim Phase 1 results for CPV-104, a first-in-class, full-length recombinant human Factor H candidate designed to treat complement-mediated diseases. Interim findings from the multiple-ascending-dose portion of the Phase 1 study demonstrated favorable safety, tolerability and pharmacokinetics, alongside encouraging early efficacy trends in patients with C3 glomerulopathy (C3G). Detailed data from the first two MAD cohorts are scheduled for presentation on October 26, 2026, following ASN Kidney Week. The company is positioning CPV-104 as a potential pipeline-in-a-product, with clinical development planned across complement-mediated kidney diseases and ophthalmic disease. One04 said its current funding is sufficient to reach several key value inflection points, including Phase 2 development in kidney disease and a new Phase 1 program in dry age-related macular degeneration (AMD) and geographic atrophy (GA).
CPV-104 Restores Endogenous Complement Regulation
CPV-104 is designed to address complement dysregulation by replacing or restoring the activity of Factor H, rather than directly inhibiting an individual component of the complement cascade. Factor H is a natural regulator of the alternative complement pathway, and One04 believes restoring its function could suppress pathological complement overactivation while preserving broader complement activity needed for immune defense and physiological homeostasis. This differentiated mechanism is central to the company’s strategy in diseases where excessive complement activation contributes to tissue damage. CPV-104 was discovered and developed using Eleva Biologics’ moss-based Bryotechnology production platform, before the asset was transferred to One04 to provide dedicated operational focus and strategic independence. The candidate has received EU Orphan Designation for C3G, supporting its development in a disease characterized by abnormal complement activation and significant unmet treatment needs.
Phase 2 Kidney Study Planned for Early 2027
One04 Therapeutics plans to initiate an open-label Phase 2 study in early 2027 evaluating CPV-104 in up to 24 patients with C3G and selected complement-mediated kidney diseases. Patients completing the Phase 1 C3G study may have an opportunity to continue treatment in the Phase 2 program, supporting longer-term assessment of safety and potential disease activity. The ongoing Phase 1 MAD portion includes three dose cohorts totaling 18 patients, with one cohort still in progress. Earlier, the placebo-controlled single-ascending-dose portion was completed in October 2025 in 21 healthy volunteers across four dose cohorts, with no safety concerns observed. The upcoming Phase 2 program is intended to build on the initial clinical evidence and further evaluate whether restoration of endogenous complement regulation can translate into meaningful benefits for patients with complement-mediated renal disorders. A larger open-label, comparator-controlled Phase 2 study is also planned for late 2027, subject to additional financing.
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One04 Expands CPV-104 Into Dry AMD and GA
The company also plans to expand CPV-104 into ophthalmology through a Phase 1 study in dry AMD and its advanced form, geographic atrophy, in early 2027. This expansion reflects the potential relevance of complement dysregulation across both kidney and eye diseases and gives One04 an opportunity to evaluate the same Factor H-based therapeutic approach across multiple indications. The company has received undisclosed funding from D11Z.Ventures to support its development strategy and reach upcoming clinical milestones. Detailed interim Phase 1 C3G data will be released through a virtual event on October 26 following ASN Kidney Week, providing a further clinical catalyst for the newly launched company. With CPV-104 advancing toward Phase 2 kidney development and a separate Phase 1 program planned for dry AMD/GA, One04 Therapeutics is building a focused complement-medicine platform around Factor H restoration and targeting multiple high-unmet-need diseases of the kidney and eye.
Source:One04 Therapeutics,, press release
